There are no FDA/EMA/etc approved therapies for mitochondrial disease. We need to work together to thoughtfully collect and integrate data to inform clinically meaningful endpoints and de-risk clinical trial design. This task force is dedicated to advancing the regulatory science needs so that individuals living with mitochondrial and inherited metabolic diseases can finally see some drug development they so desperately need and want. #missiondriven #collaboration #publicprivatepartnership #regulatory
C-Path today announced the launch of a task force focused on accelerating drug development for mitochondrial and inherited metabolic diseases. The task force will lay the groundwork for specific solutions, offering valuable insights that aim to contribute to regulatory decision-making. “C-Path is uniquely positioned to lead this new task force,” explained Amanda Klein, Pharm.D., C-Path’s Executive Director of the Transplant Therapeutics Consortium and lead for this task force. “We thank the communities for recognizing the importance of collaborative projects. We look forward to leveraging our core competencies to provide strategic and tactical guidance, engage relevant stakeholders, and bring diverse expertise to generate the solutions to help patients and their families.” Full details: https://lnkd.in/eevBsmjg Amanda Klein Alexandre Bétourné, PhD, PharmD, PMP Melody Kisor Cure LBSL Astellas Pharma Europe Dima Martini-Drew MD The Champ Foundation Cure Mito Foundation Sophia Zilber 🌺 Danielle B. mitoworld.org Alexander Sercel, PhD Midwestern University Volkmar Weissig Jon Brestoff Hope for PDCD Foundation Frances Muenzer Pimentel #CPath #taskforce #RDCADAP #NORD #FDA #raredisease #curelbsl #MitoWorld #Astellas #MitoFoundation #globalhealth #collaboration #drugdevelopment #datasharing
Executive Director at Critical Path Institute (C-Path)
8moNot to mention the increasing evidence that long COVID is a result of mitochondrial dysfunction which makes sense considering the overlapping symptoms.